- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02510261
The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)
December 5, 2023 updated by: Alnylam Pharmaceuticals
A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
Study Overview
Study Type
Interventional
Enrollment (Actual)
211
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Buenos Aires, Argentina
- Clinical Trial Site
-
-
-
-
-
Westmead, Australia
- Clinical Trial Site
-
-
-
-
-
Rio de Janeiro, Brazil
- Clinical Trial Site
-
São Paulo, Brazil
- Clinical Trial Site
-
-
-
-
-
Sofia, Bulgaria
- Clinical Trial Site
-
-
-
-
British Columbia
-
Vancouver, British Columbia, Canada
- Clinical Trial Site
-
-
-
-
-
Nicosia, Cyprus
- Clinical Trial Site
-
-
-
-
-
Creteil, France
- Clinical Trial Site
-
Le Kremlin-bicetre, France
- Clinical Trial Site
-
Lille, France
- Clinical Trial Site
-
Marseille Cedex, France
- Clinical Trial Site
-
-
Martinique
-
Fort De France, Martinique, France, 97261
- Clinical Trial Site
-
-
Reunion Island
-
Saint-Pierre, Reunion Island, France, 97448
- Clinical Trial Site
-
-
-
-
-
Cologne, Germany, 50937
- Clinical Trial Site
-
Heidelberg, Germany
- Clinical Trial Site
-
Muenster, Germany
- Clinical Trial Site
-
-
-
-
-
Pavia, Italy
- Clinical Trial Site
-
Rome, Italy
- Clinical Trial Site
-
Sicily, Italy
- Clinical Trial Site
-
-
-
-
-
Ehime, Japan
- Clinical Trial Site
-
Fukuoka, Japan
- Clinical Trial Site
-
Hiroshima, Japan
- Clinical Trial Site
-
Kumamoto, Japan
- Clinical Trial Site
-
Nagano, Japan
- Clinical Trial Site
-
Okawasuji, Japan
- Clinical Trial Site
-
Ono, Japan
- Clinical Trial Site
-
Ōita, Japan
- Clinical Trial Site
-
-
-
-
-
Seoul, Korea, Republic of
- Clinical Trial Site
-
-
-
-
-
Kuala Lumpur, Malaysia
- Clinical Trial Site
-
-
-
-
-
Tlalpan, Mexico
- Clinical Trial Site
-
-
-
-
-
Groningen, Netherlands
- Clinical Trial Site
-
-
-
-
-
Lisbon, Portugal
- Clinical Trial Site
-
Porto, Portugal
- Clinical Trial Site
-
-
-
-
-
Barcelona, Spain
- Clinical Trial Site
-
Huelva, Spain
- Clinical Trial Site
-
Madrid, Spain
- Clinical Trial Site
-
Palma De Mallorca, Spain
- Clinical Trial Site
-
-
-
-
-
Umeå, Sweden
- Clinical Trial Site
-
-
-
-
-
Taipei, Taiwan
- Clinical Trial Site
-
Taipei, Taiwan, 11217
- Clinical Trial Site
-
-
-
-
-
Istanbul, Turkey
- Clinical Trial Site
-
-
-
-
-
London, United Kingdom
- Clinical Trial Site
-
-
-
-
California
-
La Mesa, California, United States
- Clinical Trial Site
-
-
Colorado
-
Aurora, Colorado, United States
- Clinical Trial Site
-
-
Florida
-
Jacksonville, Florida, United States, 32224
- Clinical Trial Site
-
-
Illinois
-
Chicago, Illinois, United States
- Clinical Trial Site
-
-
Maryland
-
Baltimore, Maryland, United States
- Clinical Trial Site
-
-
Massachusetts
-
Boston, Massachusetts, United States
- Clinical Trial Site
-
-
Michigan
-
Detroit, Michigan, United States
- Clinical Trial Site
-
-
Minnesota
-
Rochester, Minnesota, United States
- Clinical Trial Site
-
-
Missouri
-
Joplin, Missouri, United States, 64804
- Clinical Trial Site
-
Saint Louis, Missouri, United States
- Clinical Trial Site
-
-
New York
-
Cooperstown, New York, United States
- Clinical Trial Site
-
New York, New York, United States, 10032
- Clinical Trial Site
-
New York, New York, United States, 10029
- Clinical Trial Site
-
-
Ohio
-
Columbus, Ohio, United States, 43210
- Clinical Trial Site
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 85 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug
- Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
Exclusion Criteria:
- Any new or uncontrolled condition that could make the participant unsuitable for participation
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Prior Placebo Group of Study 004
Participants who received placebo and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 milligrams per kilogram (mg/kg) patisiran intravenously (IV) once every 3 weeks (Q3W) up to 65.5 months.
|
Patisiran was administered IV.
Other Names:
|
Experimental: Prior Patisiran Group of Study 004
Participants who received patisiran and completed parent study ALN-TTR02-004 (NCT01960348) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 66.9 months.
|
Patisiran was administered IV.
Other Names:
|
Experimental: Prior Patisiran Group of Study 003
Participants who received patisiran and completed parent study ALN-TTR02-003 (NCT01961921) were enrolled to receive 0.3 mg/kg patisiran IV Q3W up to 61.4 months.
|
Patisiran was administered IV.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation
Time Frame: First dose up to 28 days after last dose of study drug (approximately 5.6 years)
|
AE is any untoward medical occurrence in a participant or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
|
First dose up to 28 days after last dose of study drug (approximately 5.6 years)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5
Time Frame: Baseline, Year 5
|
The NIS assessment is a 244-point composite measure of neurologic impairment which includes a physical exam of lower limbs, upper limbs, and cranial nerves to assess the components: motor strength/weakness (NIS-W), reflexes (NIS-R), and sensation (NIS-S).
NIS total score is obtained by combining all the component scores, ranging from 0 to 244.
Higher scores represent a greater severity of disease.
A positive change from baseline indicates the worsening of neuropathy.
|
Baseline, Year 5
|
Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3
Time Frame: Baseline, Year 3
|
The mNIS+7 is a composite measure of neurologic impairment which includes the following components: physical exam of lower limbs, upper limbs, and cranial nerves to assess motor strength/weakness (192 points), reflexes (20 points), electrophysiologic measurement of small and large nerve fiber function (10 points), sensory testing (80 points), and postural blood pressure (2 points).
The total mNIS+7 composite score is obtained by combining all the component scores, ranging from 0 (no impairment) to 304 (maximum impairment).
A negative change from baseline indicates an improvement in neuropathy.
|
Baseline, Year 3
|
Change From Baseline in the NIS+7 Total Score at Week 52
Time Frame: Baseline, Week 52
|
The NIS+7 provides additional, objective measures of nerve fibre function and autonomic nerve function in participants with diabetic neuropathy.
The NIS+7 includes the full NIS, sum of 5 nerve conduction studies (NCS) (Sural sensory nerve action potential [SNAP], tibial motor nerve distal latency, peroneal compound motor action potential [CMAP], motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing.
The total NIS+7 score is obtained by combining all the component scores, ranging from 0 (no impairment) to 270 points (maximum impairment).
A positive change from baseline indicates worsening.
|
Baseline, Week 52
|
Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5
Time Frame: Baseline, Year 5
|
The Norfolk QoL-DN questionnaire is a standardized 47-item patient-reported outcomes measure, sensitive to the perception of the effects of diabetic neuropathy by the participant.
The scores range from -4 (best possible QOL) to 136 (worst possible QOL).
A negative change from baseline represents improved QOL.
|
Baseline, Year 5
|
Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5
Time Frame: Baseline, Year 5
|
The EQ-5D-5L is a patient-reported measure of QoL based on 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression.
The overall score is rated on a scale from 0 (worst) to 1 (no impairment).
Higher scores indicate a higher QoL.
A negative change from baseline indicates worsening of QoL.
|
Baseline, Year 5
|
Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5
Time Frame: Baseline, Year 5
|
EQ-VAS measures the participant's self-rated health on a vertical scale evaluated on a scale of 0 ("worst health you can imagine") to 100 ("best health you can imagine").
Higher scores indicate a higher QOL.
A negative change from baseline indicates worsening of QoL.
|
Baseline, Year 5
|
Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52
Time Frame: Baseline, Week 52
|
COMPASS 31 questionnaire measures autonomic symptoms in participants with neuropathy.
The questionnaire consists of 31 clinically selected questions evaluating 6 autonomic domains (orthostatic intolerance, secretomotor, gastrointestinal, bladder, and pupillomotor).
COMPASS 31 is measured on a scale from 0 to 100, with 100 representing maximum impairment.
|
Baseline, Week 52
|
Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5
Time Frame: Baseline, Year 5
|
Nutritional status of participants was evaluated using the mBMI, calculated as BMI (kilograms per square meter [kg/m^2]) multiplied by the concentration of serum albumin (grams per liter [g/L]).
A positive change from baseline indicates improvement in nutritional status.
|
Baseline, Year 5
|
Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5
Time Frame: Baseline, Year 5
|
The R-ODS is a 24-item patient-reported questionnaire that specifically captures activity and social participation limitations.
It measures the level of disability on a scale of 0 (worst) to 48 (best, no limitations), higher score indicates a better outcome.
A negative change from baseline indicates worsening of disability.
|
Baseline, Year 5
|
Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5
Time Frame: Baseline, Year 5
|
The NIS+7 provides additional, objective measures of nerve fiber function and autonomic nerve function in participants with diabetic neuropathy.
The NIS+7 includes the full NIS (NIS-W, NIS-R, NIS-S), sum of 5 nerve conduction studies (NCS) (Sural SNAP, tibial motor nerve distal latency, peroneal CMAP, motor nerve conduction velocity, motor nerve distal latency), vibration detection threshold, and pulse rate response to deep breathing.
NIS-W is a measure of motor strength, comprised of cranial nerve and both upper and lower limb motor assessments.
The score ranges from 0 to 192.
A higher score indicates greater severity of disease.
|
Baseline, Year 5
|
Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5
Time Frame: Baseline, Year 5
|
10-MWT is a measure of ambulatory ability and walk speed.
It measures the speed (in meters per second [m/s]) of a participant to walk 10 meters.
A negative change from baseline represents decreased ambulatory ability.
|
Baseline, Year 5
|
Change From Baseline in the Hand Grip Strength at Week 52
Time Frame: Baseline, Week 52
|
Hand grip strength was measured by dynamometer.
Grip strength in the dominant arm is a measure of motor function, with a higher grip strength indicating better motor function.
The mean change from baseline in the hand grip strength was reported.
|
Baseline, Week 52
|
Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage
Time Frame: Baseline, Year 5
|
PND measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (sensory disturbances but preserved walking capability), II (impaired walking capability but ability to walk without a stick or crutches), IIIA (walking with help of 1 stick/crutch), IIIB (with help of 2 sticks/crutches), and IV (confined to wheelchair or bedridden).
Lower scores indicate greater ambulatory function.
The number of participants with change in the stage from baseline was reported as: Improved or worsened.
|
Baseline, Year 5
|
Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage
Time Frame: Baseline, Year 5
|
FAP measures changes in the ambulatory ability including the need of walking aids on the following stages: 0 (no symptoms), I (unimpaired ambulation; mostly mild sensory, motor, and autonomic neuropathy in the lower limbs), II (assistance with ambulation required; moderate impairment of the lower limbs, upper limbs, and trunk), and III (wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs).
Lower scores indicate greater ambulatory function.
The number of participants with change in the stage from baseline was reported as: Improved or worsened.
|
Baseline, Year 5
|
Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification
Time Frame: Baseline, Year 5
|
NYHA classification grades the severity of heart failure symptoms into the following stages: I (no symptoms; ordinary physical activity such as walking and climbing stairs does not cause fatigue or dyspnea), II (symptoms with ordinary physical activity; walking or climbing stairs rapidly, walking uphill, walking or stair climbing after meals, in cold weather, in wind or when under emotional stress causes undue fatigue or dyspnea), III (symptoms with less than ordinary physical activity; walking 1 to 2 blocks on the level and climbing more than 1 flight of stairs in normal conditions causes undue fatigue or dyspnea), IV (symptoms at rest; inability to carry on any physical activity without fatigue or dyspnea).
The number of participants with change in the stage from baseline was reported as: Improved or worsened.
|
Baseline, Year 5
|
Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5
Time Frame: Baseline, Year 5
|
IENFD (fibers/millimeter [mm]) is a measure for the pathologic evaluation of sensory and autonomic innervation.
It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg.
An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening.
|
Baseline, Year 5
|
Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5
Time Frame: Baseline, Year 5
|
SGNFD (meter/cubic millimeter [m/mm^3]) is a measure for the pathologic evaluation of sensory and autonomic innervation.
It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg.
An increase in nerve fiber density suggests improvement, while a decrease in nerve fiber density suggests worsening.
|
Baseline, Year 5
|
Change From Baseline in the Dermal Amyloid Burden at Year 5
Time Frame: Baseline, Year 5
|
Dermal Amyloid Burden is a measure for the pathologic evaluation of sensory and autonomic innervation and reported as % congo red stain.
It is obtained by tandem 3 mm skin punch biopsies: one set of biopsies taken from the distal thigh and one set from the distal lower leg.
|
Baseline, Year 5
|
Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5
Time Frame: Baseline, Year 5
|
Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: troponin (micrograms per liter [µg/L]).
The troponin I values <0.1 μg/L were imputed to 0.1 thus the actual changes cannot be calculated for values <0.1 μg/L.
|
Baseline, Year 5
|
Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5
Time Frame: Baseline, Year 5
|
Manifestations of cardiac amyloid involvement were assessed through measurement of serum levels of the cardiac biomarker: NT-proBNP (nanograms per liter [ng/L]).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of systolic function: Average peak longitudinal strain (percentage [%]).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of cardiac structure: LV mass (grams [g]).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of diastolic function: LV end-diastolic volume (milliliters [mL]).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of cardiac structure: LV relative wall thickness (ratio).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of cardiac structure: Mean LV wall thickness (centimeters [cm]).
|
Baseline, Year 5
|
Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5
Time Frame: Baseline, Year 5
|
The echocardiogram parameters analyzed included measures of systolic function: Cardiac output (liters per minute [L/min]).
|
Baseline, Year 5
|
Percent Change From Baseline in Serum TTR Levels at Year 5
Time Frame: Baseline, Year 5
|
Serum TTR was assessed using enzyme linked immunosorbent assay (ELISA).
|
Baseline, Year 5
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Alnylam Pharmaceuticals
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
July 16, 2015
Primary Completion (Actual)
November 23, 2022
Study Completion (Actual)
November 23, 2022
Study Registration Dates
First Submitted
July 16, 2015
First Submitted That Met QC Criteria
July 27, 2015
First Posted (Estimated)
July 29, 2015
Study Record Updates
Last Update Posted (Estimated)
December 6, 2023
Last Update Submitted That Met QC Criteria
December 5, 2023
Last Verified
November 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- ALN-TTR02-006
- 2014-003877-40 (EudraCT Number)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Amyloidosis
-
Millennium Pharmaceuticals, Inc.CompletedLight-Chain AmyloidosisUnited States, Canada, France, Germany, Italy
-
Boston UniversityCorino Therapeutics, Inc.CompletedTransthyretin Amyloidosis | Amyloidosis, Leptomeningeal, Transthyretin-RelatedUnited States
-
Chulalongkorn UniversityUnknown
-
Criterium, Inc.AmgenCompletedAmyloidosis | Systemic Light Chain AmyloidosisUnited States
-
Steen Hvitfeldt PoulsenRecruitingTransthyretin Amyloidosis | Transthyretin Cardiac Amyloidosis | Wild-Type Transthyretin-Related (ATTR)AmyloidosisDenmark
-
Peking Union Medical College HospitalXian-Janssen Pharmaceutical Ltd.Active, not recruitingAmyloidosis; SystemicChina
-
IRCCS Policlinico S. MatteoRecruiting
-
University Hospital Center of MartiniqueTerminated
-
Kaneka Medical America LLCRecruitingDialysis AmyloidosisUnited States
-
Peking Union Medical College HospitalRecruitingLight Chain (AL) Amyloidosis | Venetoclax | CCND1 TranslocationChina
Clinical Trials on Patisiran
-
Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis (ATTR) With CardiomyopathyUnited States, Australia, Brazil, Bulgaria, France, Italy, Japan, Mexico, Netherlands, Taiwan, United Kingdom, Belgium, Czechia, Denmark, New Zealand, Sweden, Korea, Republic of, Argentina, Chile, Hong Kong, Portugal
-
Alnylam PharmaceuticalsNo longer availableAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies
-
Austin Neuromuscular CenterAlnylam PharmaceuticalsRecruitingPolyneuropathies | Transthyretin Amyloidosis | Wild-Type Transthyretin-Related (ATTR)Amyloidosis | Wild-Type Transthyretin Cardiac Amyloidosis | Wild Type ATTR AmyloidosisUnited States
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisPortugal, Spain, Sweden, Brazil, United States, France, Germany
-
Alnylam PharmaceuticalsNo longer availableTransthyretin-mediated Amyloidosis With Cardiomyopathy | ATTR Amyloidosis With CardiomyopathyUnited States
-
Alnylam PharmaceuticalsCompletedTTR-mediated AmyloidosisPortugal, Sweden, Brazil, United States, France, Spain, Germany
-
Alnylam PharmaceuticalsCompletedA Study of the Safety, Tolerability and Pharmacokinetics of ALN-TTR02 in Japanese Healthy VolunteersTransthyretin (TTR)-Mediated AmyloidosisUnited Kingdom
-
Alnylam PharmaceuticalsCompletedAmyloidosis, Familial | Transthyretin AmyloidosisFrance, Germany, Italy, Portugal, Spain, Sweden, United Kingdom
-
Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis | Amyloidosis, HereditaryUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
-
Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom